CA2949697A1 - Procede d'edition d'une sequence genetique - Google Patents

Procede d'edition d'une sequence genetique Download PDF

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Publication number
CA2949697A1
CA2949697A1 CA2949697A CA2949697A CA2949697A1 CA 2949697 A1 CA2949697 A1 CA 2949697A1 CA 2949697 A CA2949697 A CA 2949697A CA 2949697 A CA2949697 A CA 2949697A CA 2949697 A1 CA2949697 A1 CA 2949697A1
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CA
Canada
Prior art keywords
sequence
cell
fancc
cells
donor
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
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CA2949697A
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English (en)
Inventor
Jakub Tolar
Bruce Robert Blazar
Daniel Francis Voytas
Mark John Osborn
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University of Minnesota
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University of Minnesota
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Publication date
Application filed by University of Minnesota filed Critical University of Minnesota
Publication of CA2949697A1 publication Critical patent/CA2949697A1/fr
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La présente invention concerne des procédés, des polynucléotides, des cellules, des compositions et des méthodes de traitement qui consistent à modifier une séquence nucléotidique génomique. Généralement, le procédé consiste à introduire, dans une cellule possédant une séquence génomique devant être éditée, le polynucléotide d'un donneur et un nucléotide codant pour une enzyme capable de découper au moins un brin d'ADN, à laisser l'enzyme découper au moins un brin de la séquence génomique et à laisser la séquence du donneur remplacer la séquence génomique devant être éditée.
CA2949697A 2014-05-20 2015-05-20 Procede d'edition d'une sequence genetique Abandoned CA2949697A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462000590P 2014-05-20 2014-05-20
US62/000,590 2014-05-20
PCT/US2015/031807 WO2015179540A1 (fr) 2014-05-20 2015-05-20 Procédé d'édition d'une séquence génétique

Publications (1)

Publication Number Publication Date
CA2949697A1 true CA2949697A1 (fr) 2015-11-26

Family

ID=54554709

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2949697A Abandoned CA2949697A1 (fr) 2014-05-20 2015-05-20 Procede d'edition d'une sequence genetique

Country Status (5)

Country Link
US (1) US20170175143A1 (fr)
EP (1) EP3152221A4 (fr)
JP (1) JP2017517256A (fr)
CA (1) CA2949697A1 (fr)
WO (1) WO2015179540A1 (fr)

Families Citing this family (38)

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EP3613852A3 (fr) 2011-07-22 2020-04-22 President and Fellows of Harvard College Évaluation et amélioration de la spécificité de clivage des nucléases
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
MX2016010285A (es) 2014-02-11 2017-01-11 Univ Colorado Regents Ingenieria genetica multiplexada habilitada por crispr.
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3294896A1 (fr) * 2015-05-11 2018-03-21 Editas Medicine, Inc. Systèmes crispr/cas9 optimisés et procédés d'édition de gènes dans des cellules souches
AU2016276702B2 (en) 2015-06-09 2022-07-28 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for improving transplantation
CN108513575A (zh) 2015-10-23 2018-09-07 哈佛大学的校长及成员们 核碱基编辑器及其用途
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
WO2018027078A1 (fr) 2016-08-03 2018-02-08 President And Fellows Of Harard College Éditeurs de nucléobases d'adénosine et utilisations associées
CA3033327A1 (fr) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Proteines de fusion cas9-recombinase programmables et utilisations associees
WO2018030457A1 (fr) * 2016-08-10 2018-02-15 武田薬品工業株式会社 Procédé de modification d'un site cible dans le génome d'une cellule eucaryote, et procédé de détection de la présence ou de l'absence d'une séquence d'acide nucléique à détecter au niveau d'un site cible
WO2018039438A1 (fr) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation d'acides aminés non naturels dans des protéines au moyen de l'édition de bases
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR101997116B1 (ko) 2016-10-14 2019-07-05 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
WO2018070850A1 (fr) * 2016-10-14 2018-04-19 연세대학교 산학협력단 Arn guide complémentaire du gène kras, et son utilisation
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression de la douleur par édition de gène
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
SG11201908658TA (en) 2017-03-23 2019-10-30 Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
EP3652312A1 (fr) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systèmes et procédés d'intégration ciblée et d'édition du génome et détection de celle-ci à l'aide de sites d'amorçage intégrés
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
MX2020001177A (es) 2017-07-31 2020-09-25 Regeneron Pharma Animales no humanos reporteros de crispr y usos de los mismos.
SG11201912235PA (en) 2017-07-31 2020-01-30 Regeneron Pharma Cas-transgenic mouse embryonic stem cells and mice and uses thereof
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
BR112020020841A2 (pt) * 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CN113454235A (zh) * 2019-02-21 2021-09-28 豪夫迈·罗氏有限公司 经改进的核酸靶标富集和相关方法
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
EP4146804A1 (fr) 2020-05-08 2023-03-15 The Broad Institute Inc. Méthodes et compositions d'édition simultanée des deux brins d'une séquence nucléotidique double brin cible

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Publication number Priority date Publication date Assignee Title
US20060228798A1 (en) * 2002-11-27 2006-10-12 Catherine Verfaillie Homologous recombination in multipotent adult progenitor cells
DK3401400T3 (da) * 2012-05-25 2019-06-03 Univ California Fremgangsmåder og sammensætninger til rna-styret mål-dna-modifikation og til rna-styret transskriptionsmodulering
EP3763810A3 (fr) * 2012-10-10 2021-07-14 Sangamo Therapeutics, Inc. Composés de modification de cellules t et leurs utilisations
CN110643600A (zh) * 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的***及其用途

Also Published As

Publication number Publication date
JP2017517256A (ja) 2017-06-29
US20170175143A1 (en) 2017-06-22
WO2015179540A1 (fr) 2015-11-26
EP3152221A1 (fr) 2017-04-12
EP3152221A4 (fr) 2018-01-24

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Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20211123

FZDE Discontinued

Effective date: 20211123